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Intrathecal (G4C2)149 delivery in C9orf72-deficient mice yields mild motor dysfunction and ALS/FTD pathological hallmarks.

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Title: Intrathecal (G4C2)149 delivery in C9orf72-deficient mice yields mild motor dysfunction and ALS/FTD pathological hallmarks.
Authors: Russell KA; Department of Neuroscience, Vickie and Jack Farber Institute for Neuroscience, Thomas Jefferson University, Philadelphia, PA 19107, USA.; Jefferson Weinberg ALS Center, Vickie and Jack Farber Institute for Neuroscience, Thomas Jefferson University, Philadelphia, PA 19107, USA., Shahrabi AA; Department of Neuroscience, Vickie and Jack Farber Institute for Neuroscience, Thomas Jefferson University, Philadelphia, PA 19107, USA.; Jefferson Weinberg ALS Center, Vickie and Jack Farber Institute for Neuroscience, Thomas Jefferson University, Philadelphia, PA 19107, USA., Akerman SC; Department of Neurology, Johns Hopkins University School of Medicine, Baltimore, MD, 21205, USA.; Brain Science Institute, Johns Hopkins University School of Medicine, Baltimore, MD, 21205, USA., Byrne MD; Department of Neuroscience, Vickie and Jack Farber Institute for Neuroscience, Thomas Jefferson University, Philadelphia, PA 19107, USA., Rothstein JD; Department of Neurology, Johns Hopkins University School of Medicine, Baltimore, MD, 21205, USA.; Brain Science Institute, Johns Hopkins University School of Medicine, Baltimore, MD, 21205, USA., Trotti D; Department of Neuroscience, Vickie and Jack Farber Institute for Neuroscience, Thomas Jefferson University, Philadelphia, PA 19107, USA.; Jefferson Weinberg ALS Center, Vickie and Jack Farber Institute for Neuroscience, Thomas Jefferson University, Philadelphia, PA 19107, USA., Jensen BK; Department of Neuroscience, Vickie and Jack Farber Institute for Neuroscience, Thomas Jefferson University, Philadelphia, PA 19107, USA.; Jefferson Weinberg ALS Center, Vickie and Jack Farber Institute for Neuroscience, Thomas Jefferson University, Philadelphia, PA 19107, USA., Haeusler AR; Department of Neuroscience, Vickie and Jack Farber Institute for Neuroscience, Thomas Jefferson University, Philadelphia, PA 19107, USA.; Jefferson Weinberg ALS Center, Vickie and Jack Farber Institute for Neuroscience, Thomas Jefferson University, Philadelphia, PA 19107, USA.
Source: BioRxiv : the preprint server for biology [bioRxiv] 2026 Feb 11. Date of Electronic Publication: 2026 Feb 11.
Publication Type: Journal Article; Preprint
Journal Info: Country of Publication: United States NLM ID: 101680187 Publication Model: Electronic Cited Medium: Internet ISSN: 2692-8205 (Electronic) Linking ISSN: 26928205 NLM ISO Abbreviation: bioRxiv Subsets: PubMed not MEDLINE
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  Data: Intrathecal (G<subscript>4</subscript>C<subscript>2</subscript>)<subscript>149</subscript> delivery in C9orf72-deficient mice yields mild motor dysfunction and ALS/FTD pathological hallmarks.
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  Data: <searchLink fieldCode="AU" term="%22Russell+KA%22">Russell KA</searchLink>; Department of Neuroscience, Vickie and Jack Farber Institute for Neuroscience, Thomas Jefferson University, Philadelphia, PA 19107, USA.; Jefferson Weinberg ALS Center, Vickie and Jack Farber Institute for Neuroscience, Thomas Jefferson University, Philadelphia, PA 19107, USA.<br /><searchLink fieldCode="AU" term="%22Shahrabi+AA%22">Shahrabi AA</searchLink>; Department of Neuroscience, Vickie and Jack Farber Institute for Neuroscience, Thomas Jefferson University, Philadelphia, PA 19107, USA.; Jefferson Weinberg ALS Center, Vickie and Jack Farber Institute for Neuroscience, Thomas Jefferson University, Philadelphia, PA 19107, USA.<br /><searchLink fieldCode="AU" term="%22Akerman+SC%22">Akerman SC</searchLink>; Department of Neurology, Johns Hopkins University School of Medicine, Baltimore, MD, 21205, USA.; Brain Science Institute, Johns Hopkins University School of Medicine, Baltimore, MD, 21205, USA.<br /><searchLink fieldCode="AU" term="%22Byrne+MD%22">Byrne MD</searchLink>; Department of Neuroscience, Vickie and Jack Farber Institute for Neuroscience, Thomas Jefferson University, Philadelphia, PA 19107, USA.<br /><searchLink fieldCode="AU" term="%22Rothstein+JD%22">Rothstein JD</searchLink>; Department of Neurology, Johns Hopkins University School of Medicine, Baltimore, MD, 21205, USA.; Brain Science Institute, Johns Hopkins University School of Medicine, Baltimore, MD, 21205, USA.<br /><searchLink fieldCode="AU" term="%22Trotti+D%22">Trotti D</searchLink>; Department of Neuroscience, Vickie and Jack Farber Institute for Neuroscience, Thomas Jefferson University, Philadelphia, PA 19107, USA.; Jefferson Weinberg ALS Center, Vickie and Jack Farber Institute for Neuroscience, Thomas Jefferson University, Philadelphia, PA 19107, USA.<br /><searchLink fieldCode="AU" term="%22Jensen+BK%22">Jensen BK</searchLink>; Department of Neuroscience, Vickie and Jack Farber Institute for Neuroscience, Thomas Jefferson University, Philadelphia, PA 19107, USA.; Jefferson Weinberg ALS Center, Vickie and Jack Farber Institute for Neuroscience, Thomas Jefferson University, Philadelphia, PA 19107, USA.<br /><searchLink fieldCode="AU" term="%22Haeusler+AR%22">Haeusler AR</searchLink>; Department of Neuroscience, Vickie and Jack Farber Institute for Neuroscience, Thomas Jefferson University, Philadelphia, PA 19107, USA.; Jefferson Weinberg ALS Center, Vickie and Jack Farber Institute for Neuroscience, Thomas Jefferson University, Philadelphia, PA 19107, USA.
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  Data: <searchLink fieldCode="JN" term="%22101680187%22">BioRxiv : the preprint server for biology</searchLink> [bioRxiv] 2026 Feb 11. <i>Date of Electronic Publication: </i>2026 Feb 11.
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        Value: 10.64898/2026.02.09.704060
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